Desai discusses the potential long-term effects of mavacamten and what it could mean for a patient population with HCM.
The administration of mavacamten was found to significantly reduce eligibility for invasive septal reduction therapy (SRT) at 16 weeks in patients with obstructive hypertrophic cardiomyopathy (HCM).
The late-breaking findings were presented at The American College of Cardiology (ACC) 2022 Scientific Sessions in Washington, DC.
In an interview with HCPLive, principal investigator Milind Desai, MD, Cleveland Clinic, discussed what these results meant for patients with HCM and how they might transform the treamtment of the condition going forward.
He highlighted the first-in-class agent is aiming to address the unmet need of precision, not invasive medical therapies in patients with HCM. As a 16 week trial, questions remain on both maintaining efficacy long-term and any potential safety signals.
Desai additionally noted the different contingencies for patients, as in some this may delay septal reduction therapy (SRT) for a time, while others may not need any further procedures. Still, others are not candidates for the surgery due to a much higher risk.
"The last important thing is there is a large group of population on this planet who may not have access to high quality specialized invasive care," Desai said.
He went on to discuss the tremendous population with HCM being a long-term issue, as patients may be undiagnosed, underdiagnosed, or misdiagnosed without access to proper care. However, he noted that imaging is improving as is risk stratification.
Lastly, Desai noted that at 16 weeks, patients were offered SRT or to continue using mavacamten, with 95% of patients continuing medical therapy. As a result, 32-week data will aid in determining safety signals, eligibility, and guidelines.
"Science should always override narratives, you know, a randomized control trial with a difficult trial design like this is difficult to do in the context of a COVID pandemic," Desai said. "Is it impossible? No. We've shown it that you know, if we put the right minds and collective efforts behind something, we can do whatever we want."